Myelodysplastic Syndrome Treatment by Ray Sahelian, M.D.

Treatment of patients with a myelodysplastic syndrome (MDS) is challenging. The dilemma that confronts the management of myelodysplastic syndrome is illustrated by the availability of two drugs (5-azacitidine and decitabine) with an indication for all subtypes of this disease and another one (lenalidomide) for the management of a specific myelodysplastic syndrome subgroup, the 5q- syndrome. Current classifications and prognostic systems do not take into account the considerable clinical heterogeneity of myelodysplastic syndrome or their diverse biology. Supportive care, low-intensity treatment, acute myeloid leukemia-type therapy, and stem cell transplantation (SCT) produce unsatisfactory results because patients continue to be exposed to the inherent complications of worsening cytopenias and leukemic transformation. Recent years have witnessed an evolution in our understanding of pathophysiology pathways in myelodysplastic syndrome. At the same time, many novel and targeted therapies are being investigated in clinical trials, offering patients the prospect of sustained benefit and changing the natural course of the disease. Hypomethylating agents, immunomodulatory drugs, and farnesyl-tranferase inhibitors have produced very promising results in terms of response and survival in myelodysplastic syndrome patients.

Myelodysplastic syndrome and Decitabine
Dacogen (decitabine, MGI Pharma) for Injection is available for the treatment of patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo, and secondary myelodysplastic syndrome of all French-American-British (FAB) subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia), and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System (IPSS) groups.